Chronic Inflammatory Demyelinating Polyradiculoneuropathy: Current Therapeutic Approaches and Future Outlooks.

Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a treatable autoimmune disorder, for which different treatment options are available. Current first-line evidence-based therapies for CIDP include intravenous and subcutaneous immunoglobulins, corticosteroids and plasma exchanges. Despite lack of evidence, cyclophosphamide, rituximab and mycophenolate mofetil are commonly used in circumstances of refractoriness and, more debatably, of perceived overdependence on first-line therapies. Rituximab is currently the object of a randomized controlled trial for CIDP. Based on case series, and although rarely considered, haematopoietic autologous stem cell transplants may be effective in refractory disease, with low mortality and high remission rates. A new therapeutic option has appeared with efgartigimod, a neonatal Fc receptor blocker, recently shown to significantly lower relapse rate versus placebo, after withdrawal from previous immunotherapy. Other neonatal Fc receptor blockers, nipocalimab and batoclimab, are under study. The C1 complement-inhibitor SAR445088, acting in the proximal portion of the classical complement system, is currently the subject of a new study in treatment-responsive, refractory and treatment-naïve subjects. Finally, Bruton Tyrosine Kinase inhibitors, which exert anti-B cell effects, may represent another future research avenue. The widening of the therapeutic armamentarium enhances the need for improved evaluation of treatment effects and reliable biomarkers in CIDP.

Impact of social-functioning and sleep on quality of life in chronic inflammatory demyelinating polyneuropathy.

INTRODUCTION/AIMS: The impact of impairment of social functioning and sleep on health-related quality of life (HR-QoL), is unknown in chronic inflammatory demyelinating polyneuropathy (CIDP). The value of the Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI) to identify potential social functioning and sleep issues is equally unknown. METHODS: We performed a cross-sectional evaluation of social functioning and sleep using the "Scales for Outcomes in Parkinson's Disease" (SCOPA) in 40 subjects with clinically-stable CIDP through a structured questionnaire. We assessed HR-QoL through the CAP-PRI. Disability was evaluated through the Overall Neuropathy Limitation Score (ONLS). RESULTS: SCOPA social functioning scores were impaired at least "a little" per averaged item in > 50 % of subjects, and at least "quite a bit" per averaged item in > 20 %. Most affected items were (i) difficulty with work/household/other chores (ii) difficulties with hobbies/sport/leisure activities. SCOPA sleep sub-scores indicated at least "a little concern" for night-time sleep in nearly 50 % of subjects. Abnormal sleep timing was rare. Associations were found between both SCOPA social-functioning and SCOPA sleep scores and the CAP-PRI. Linear regression demonstrated the SCOPA social-functioning score was independently associated with the CAP-PRI. The CAP-PRI showed high association with disability scores, good internal consistency, absence of ceiling effect, absence of significant floor-effect, and good criteria-related as well as construct-related validity. DISCUSSION: Social functioning and night-time sleep are frequently affected in CIDP and impact on HR-QoL. In contrast to traditional disability scales, the CAP-PRI additionally allows adequately capturing these impairments and may represent an adequate holistic outcome measure.

European Academy of Neurology/Peripheral Nerve Society Guideline on diagnosis and treatment of Guillain-Barré syndrome.

Guillain-Barré syndrome (GBS) is an acute polyradiculoneuropathy. Symptoms may vary greatly in presentation and severity. Besides weakness and sensory disturbances, patients may have cranial nerve involvement, respiratory insufficiency, autonomic dysfunction and pain. To develop an evidence-based guideline for the diagnosis and treatment of GBS, using Grading of Recommendations, Assessment, Development and Evaluation (GRADE) methodology, a Task Force (TF) of the European Academy of Neurology (EAN) and the Peripheral Nerve Society (PNS) constructed 14 Population/Intervention/Comparison/Outcome questions (PICOs) covering diagnosis, treatment and prognosis of GBS, which guided the literature search. Data were extracted and summarised in GRADE Summaries of Findings (for treatment PICOs) or Evidence Tables (for diagnostic and prognostic PICOs). Statements were prepared according to GRADE Evidence-to-Decision (EtD) frameworks. For the six intervention PICOs, evidence-based recommendations are made. For other PICOs, good practice points (GPPs) are formulated. For diagnosis, the principal GPPs are: GBS is more likely if there is a history of recent diarrhoea or respiratory infection; CSF examination is valuable, particularly when the diagnosis is less certain; electrodiagnostic testing is advised to support the diagnosis; testing for anti-ganglioside antibodies is of limited clinical value in most patients with typical motor-sensory GBS, but anti-GQ1b antibody testing should be considered when Miller Fisher syndrome (MFS) is suspected; nodal-paranodal antibodies should be tested when autoimmune nodopathy is suspected; MRI or ultrasound imaging should be considered in atypical cases; and changing the diagnosis to acute-onset chronic inflammatory demyelinating polyradiculoneuropathy (A-CIDP) should be considered if progression continues after 8 weeks from onset, which occurs in around 5% of patients initially diagnosed with GBS. For treatment, the TF recommends intravenous immunoglobulin (IVIg) 0.4 g/kg for 5 days, in patients within 2 weeks (GPP also within 2-4 weeks) after onset of weakness if unable to walk unaided, or a course of plasma exchange (PE) 12-15 L in four to five exchanges over 1-2 weeks, in patients within 4 weeks after onset of weakness if unable to walk unaided. The TF recommends against a second IVIg course in GBS patients with a poor prognosis; recommends against using oral corticosteroids, and weakly recommends against using IV corticosteroids; does not recommend PE followed immediately by IVIg; weakly recommends gabapentinoids, tricyclic antidepressants or carbamazepine for treatment of pain; does not recommend a specific treatment for fatigue. To estimate the prognosis of individual patients, the TF advises using the modified Erasmus GBS outcome score (mEGOS) to assess outcome, and the modified Erasmus GBS Respiratory Insufficiency Score (mEGRIS) to assess the risk of requiring artificial ventilation. Based on the PICOs, available literature and additional discussions, we provide flow charts to assist making clinical decisions on diagnosis, treatment and the need for intensive care unit admission.

European Academy of Neurology/Peripheral Nerve Society Guideline on diagnosis and treatment of Guillain-Barré syndrome.

Guillain-Barré syndrome (GBS) is an acute polyradiculoneuropathy. Symptoms may vary greatly in presentation and severity. Besides weakness and sensory disturbances, patients may have cranial nerve involvement, respiratory insufficiency, autonomic dysfunction and pain. To develop an evidence-based guideline for the diagnosis and treatment of GBS, using Grading of Recommendations, Assessment, Development and Evaluation (GRADE) methodology a Task Force (TF) of the European Academy of Neurology (EAN) and the Peripheral Nerve Society (PNS) constructed 14 Population/Intervention/Comparison/Outcome questions (PICOs) covering diagnosis, treatment and prognosis of GBS, which guided the literature search. Data were extracted and summarised in GRADE Summaries of Findings (for treatment PICOs) or Evidence Tables (for diagnostic and prognostic PICOs). Statements were prepared according to GRADE Evidence-to-Decision (EtD) frameworks. For the six intervention PICOs, evidence-based recommendations are made. For other PICOs, good practice points (GPPs) are formulated. For diagnosis, the principal GPPs are: GBS is more likely if there is a history of recent diarrhoea or respiratory infection; CSF examination is valuable, particularly when the diagnosis is less certain; electrodiagnostic testing is advised to support the diagnosis; testing for anti-ganglioside antibodies is of limited clinical value in most patients with typical motor-sensory GBS, but anti-GQ1b antibody testing should be considered when Miller Fisher syndrome (MFS) is suspected; nodal-paranodal antibodies should be tested when autoimmune nodopathy is suspected; MRI or ultrasound imaging should be considered in atypical cases; and changing the diagnosis to acute-onset chronic inflammatory demyelinating polyradiculoneuropathy (A-CIDP) should be considered if progression continues after 8 weeks from onset, which occurs in around 5% of patients initially diagnosed with GBS. For treatment, the TF recommends intravenous immunoglobulin (IVIg) 0.4 g/kg for 5 days, in patients within 2 weeks (GPP also within 2-4 weeks) after onset of weakness if unable to walk unaided, or a course of plasma exchange (PE) 12-15 L in four to five exchanges over 1-2 weeks, in patients within 4 weeks after onset of weakness if unable to walk unaided. The TF recommends against a second IVIg course in GBS patients with a poor prognosis; recommends against using oral corticosteroids, and weakly recommends against using IV corticosteroids; does not recommend PE followed immediately by IVIg; weakly recommends gabapentinoids, tricyclic antidepressants or carbamazepine for treatment of pain; does not recommend a specific treatment for fatigue. To estimate the prognosis of individual patients, the TF advises using the modified Erasmus GBS outcome score (mEGOS) to assess outcome, and the modified Erasmus GBS Respiratory Insufficiency Score (mEGRIS) to assess the risk of requiring artificial ventilation. Based on the PICOs, available literature and additional discussions, we provide flow charts to assist making clinical decisions on diagnosis, treatment and the need for intensive care unit admission.

Advances in diagnosis and management of distal sensory polyneuropathies.

Distal sensory polyneuropathy (DSP) is characterised by length-dependent, sensory-predominant symptoms and signs, including potentially disabling symmetric chronic pain, tingling and poor balance. Some patients also have or develop dysautonomia or motor involvement depending on whether large myelinated or small fibres are predominantly affected. Although highly prevalent, diagnosis and management can be challenging. While classic diabetes and toxic causes are well-recognised, there are increasingly diverse associations, including with dysimmune, rheumatological and neurodegenerative conditions. Approximately half of cases are initially considered idiopathic despite thorough evaluation, but often, the causes emerge later as new symptoms develop or testing advances, for instance with genetic approaches. Improving and standardising DSP metrics, as already accomplished for motor neuropathies, would permit in-clinic longitudinal tracking of natural history and treatment responses. Standardising phenotyping could advance research and facilitate trials of potential therapies, which lag so far. This review updates on recent advances and summarises current evidence for specific treatments.

Application of the 2021 EAN/PNS criteria for chronic inflammatory demyelinating polyneuropathy.

BACKGROUND: The diagnostic value of new criteria of the European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) for chronic inflammatory demyelinating polyneuropathy (CIDP) is unknown. METHODS: We performed a retrospective study of fulfilment of EAN/PNS 2021 criteria on 120 consecutive patients with a clinical diagnosis of 'suspected CIDP' and objective treatment response, attending University Hospitals Birmingham, UK. Specificity was evaluated versus 100 consecutive controls. RESULTS: The sensitivity of EAN/PNS criteria for 'CIDP' was 83.3%. The sensitivity for 'CIDP' or 'possible CIDP' was 93.3%. Specificity was of 94% for 'CIDP' and 79% for 'CIDP' or 'possible CIDP'. No sensitivity/specificity differences were ascertained with previous versions ('CIDP': sensitivity: 83.3% vs 81.3%, p=0.74, specificity: 94% vs 96.1%, p=0.38, respectively; 'CIDP' or 'possible CIDP': sensitivity: 93.3% vs 96.7%, p=0.25 and specificity: 79% vs 69.2 %, p=0.09, respectively). F-wave prolongation, proximal and distal temporal dispersion were the most likely parameters to contribute to false positives, whereas distal motor latency was the least likely. No impact of sensory electrophysiology could be ascertained. 'Typical CIDP' represented 79% of the CIDP cohort. The largest component of the 'variant CIDP' group was represented by focal/multifocal forms (14%). With new criteria, 6.7% of the cohort did not meet requirements, among whom the majority (75%) had paranodopathy or chronic immune sensory polyradiculopathy (CISP). DISCUSSION: The sensitivity and specificity of new EAN/PNS criteria for CIDP is equivalent to that of previous versions. The exclusion of paranodopathies and CISP from the CIDP spectrum impacts on management of a non-negligible proportion of treatment-responsive patients.

Assessment timing and choice of outcome measure in determining treatment response in chronic inflammatory demyelinating polyneuropathy: A post hoc analysis of the PRISM trial.

INTRODUCTION/AIMS: Treatment response and its timing are variable in chronic inflammatory demyelinating polyneuropathy (CIDP). In this study we assessed the variability using multiple outcome measures. METHODS: We performed a post hoc analysis of the PRISM trial, a 24-week prospective, multicenter, single-arm, open-label, phase III study of a 10% intravenous immunoglobulin preparation for CIDP. We ascertained timing of response with primary/secondary outcome measures. RESULTS: At 6 weeks after treatment initiation, 13 of 40 subjects (32.5%) were defined as responders on the primary outcome measure, the adjusted Inflammatory Neuropathy Cause And Treatment (INCAT) scale. This increased to 20 of 41 (48.8%) at 12 weeks and to 32 of 42 (76.2%) at 24 weeks. Use of minimal important difference (MID)-determined amelioration of the inflammatory Rasch-built Overall Disability Scale (I-RODS), or of the Medical Research Council sum score (MRCSS), or of dominant hand-grip strength, in addition to the adjusted INCAT, indicated a sensitivity of 41.7% in identifying adjusted INCAT nonresponders at week 12 who subsequently responded at week 24. Specificity was 60% vs INCAT nonresponders at week 24. Consideration of amelioration of any amplitude on any secondary outcome measure indicated a 75% sensitivity, but only 30% specificity vs adjusted INCAT nonresponders at week 24. DISCUSSION: Immunoglobulin treatment continuation may be justified for up to 24 weeks in CIDP. Additional outcome measures may help in the early treatment stages to predict delayed response on the adjusted INCAT. However, their use is limited by high false-positive rates. More robust, reliable, and relevant outcome measures are needed to detect early improvement in immunoglobulin-treated CIDP.

Immunoglobulin and Monoclonal Antibody Therapies in Guillain-Barré Syndrome.

Guillain-Barré syndrome (GBS) is an acute autoimmune polyradiculoneuropathy affecting 1-2 subjects per 100,000 every year worldwide. It causes, in its classic form, symmetric weakness in the proximal and distal limb muscles with common involvement of the cranial nerves, particularly facial weakness. Respiratory function is compromised in a case in four. Randomised controlled trials have demonstrated the benefit of therapeutic plasma exchange in hastening time to recovery. Intravenous immunoglobulin was subsequently shown to be as efficacious as plasma exchange in adult subjects. In children, few trials have shown the benefit of intravenous immunoglobulin versus supportive care. Pharmacokinetic studies suggested a relationship between increase in immunoglobulin G level post-infusion and outcome, implying administration of larger doses may be beneficial in subjects with poor prognosis. However, a subsequent trial of a second dose of immunoglobulin in such subjects failed to show improved outcome, while demonstrating a higher risk of thromboembolic side-effects. Monoclonal antibody therapy has more recently been investigated for GBS, after multiple studies in animal models, with different agents and variable postulated mechanisms of action. Eculizumab, a humanised monoclonal antibody against the complement protein C5, was tested in in two randomised, double-blind, placebo-controlled phase 2 trials. Neither showed benefit versus immunoglobulins alone on disability level at 4 weeks, although one study importantly suggested possible, clinically highly relevant, late effects on normalising function. A phase 3 trial is in progress. Preliminary results of a placebo-controlled ongoing study of ANX005, a humanised recombinant antibody against C1q inhibiting the complement cascade, have been promising.

Contemporary challenges in the diagnosis and management of chronic inflammatory demyelinating polyneuropathy.

INTRODUCTION: Despite extensive research, multiple inter-related diagnostic and management challenges remain for chronic inflammatory demyelinating polyneuropathy (CIDP). AREAS COVERED: A literature review was performed on diagnosis and treatment in CIDP. The clinical features and disease course were evaluated. Investigative techniques, including electrophysiology, cerebrospinal fluid examination, neuropathology, imaging, and neuroimmunology, were considered in relation to technical aspects, sensitivity, specificity, availability, and cost. Available evidenced-based treatments and those with possible efficacy despite lack of evidence, were considered, as well as current methods for evaluation of treatment effects. EXPERT OPINION: CIDP remains a clinical diagnosis, supported first and foremost by electrophysiology. Other investigative techniques have limited impact. Most patients with CIDP respond to available first-line treatments and immunosuppression may be efficacious in those who do not. Consideration of the natural history and of the high reported remission rate, of under-recognized associated disabling features, of treatment administration modalities and assessment methods, require enhanced attention.

The value of MUNIX as an objective electrophysiological biomarker of disease progression in chronic inflammatory demyelinating polyneuropathy.

INTRODUCTION/AIMS: Objective outcome measures to monitor treatment response and guide treatment are lacking in chronic inflammatory demyelinating polyneuropathy (CIDP). In this study we aimed to evaluate the motor unit number index (MUNIX) as an outcome measurement in patients with CIDP and determine the correlation of MUNIX with functional and standard electrodiagnostic tests in a single follow-up study. METHODS: We evaluated MUNIX of the abductor pollicis brevis, abductor digiti minimi, and tibialis anterior (TA) muscles bilaterally. Muscle force was assessed by Medical Research Council Sum Score (MRCSS). Functional measures used were the Overall Neuropathy Limitation Score (ONLS) and the Rasch-built Overall Disability Scale (R-ODS) score at baseline and after 6 months of treatment. Standard electrophysiology was evaluated by the Nerve Conduction Study Score (NCSS). RESULTS: Twenty patients were included at baseline, and 16 completed the follow-up study. Significant correlations were found between the MUNIX sum score and both MRCSS and NCSS at baseline, between both the pinch strength and grip and upper limb MUNIX at baseline and follow-up, and between MUNIX of TA and both lower limb MRCSSs with lower limb ONLS at baseline and follow-up. Significant correlations also were found between MUNIX sum score change and MRCSS change, R-ODS change, and ONLS change. DISCUSSION: MUNIX changes correlated with strength and electrophysiological improvements in CIDP patients. This suggests that MUNIX may represent a useful objective biomarker for patient follow-up.

Evidence base for investigative and therapeutic modalities in chronic inflammatory demyelinating polyneuropathy and multifocal motor neuropathy.

Chronic inflammatory demyelinating polyneuropathy, its variants and multifocal motor neuropathy belong to a spectrum of peripheral nerve disorders with complex dysimmune disease mechanisms. Awareness of the unique clinical phenotypes but also heterogeneity between patients is vital to arrive at early suspicion and ordering appropriate tests. This includes requirements for optimal electrodiagnostic protocol, aimed to capture sufficient electrophysiologic evidence for relevant abnormalities, a case-based approach on the eventual need to further expand the diagnostic armamentarium and correct reading of their results. Considerable phenotypical variation, diverse combinations of abnormalities found on diagnostic tests and heterogeneity in disease course and treatment response, all contribute to widespread differences in success rates on timely diagnosis and optimal treatment. We aim to provide a practical overview and guidance on relevant diagnostic and management strategies, including pitfalls and present a summary of the relevant novel developments in this field.

Electrodiagnosis of Guillain-Barre syndrome in the International GBS Outcome Study: Differences in methods and reference values.

OBJECTIVE: To describe the heterogeneity of electrodiagnostic (EDx) studies in Guillain-Barré syndrome (GBS) patients collected as part of the International GBS Outcome Study (IGOS). METHODS: Prospectively collected clinical and EDx data were available in 957 IGOS patients from 115 centers. Only the first EDx study was included in the current analysis. RESULTS: Median timing of the EDx study was 7 days (interquartile range 4-11) from symptom onset. Methodology varied between centers, countries and regions. Reference values from the responding 103 centers were derived locally in 49%, from publications in 37% and from a combination of these in the remaining 15%. Amplitude measurement in the EDx studies (baseline-to-peak or peak-to-peak) differed from the way this was done in the reference values, in 22% of motor and 39% of sensory conduction. There was marked variability in both motor and sensory reference values, although only a few outliers accounted for this. CONCLUSIONS: Our study showed extensive variation in the clinical practice of EDx in GBS patients among IGOS centers across the regions. SIGNIFICANCE: Besides EDx variation in GBS patients participating in IGOS, this diversity is likely to be present in other neuromuscular disorders and centers. This underlines the need for standardization of EDx in future multinational GBS studies.

Causes and consequences of diagnostic delay in Guillain-Barré syndrome in a UK tertiary center.

INTRODUCTION/AIMS: Understanding the potential causes and consequences of diagnostic delay in Guillain-Barré syndrome (GBS) could improve quality of care and outcomes. We aimed to determine these causes and consequences in our cohort of patients with GBS. METHODS: We retrospectively reviewed records of subjects with GBS, admitted to our center at University Hospitals Birmingham, UK, between January 2005 and December 2020. We evaluated time to diagnosis from presentation, factors associated with diagnostic delay, and its potential consequences. RESULTS: We included 119 consecutive subjects. Diagnostic delay at least 5 days from first presentation occurred in 27 of 119 (22.7%) of patients. Diagnostic delay was associated with age >60 years (odds ratio [OR], 3.58; 95% confidence interval [CI], 1.44-8.85), pre-existing cardiac/respiratory disease (OR, 4.10; 95% CI, 1.46-11.54), pre-existing diabetes (OR, 10.38; 95% CI, 2.47-43.69), documented normal initial neurological examination (OR, 2.49; 95% CI, 1.03-6.02), initial assessment by primary care (OR, 3.33; 95% CI, 1.22-9.10) and at least one visit for medical attention (OR, 10.29; 95% CI, 3.81-27.77). Diagnostic delay was not associated with length of inpatient stay, intensive care unit admission, ventilation, ability to walk at discharge, or inpatient mortality. Independent associations with diagnostic delay were observed for at least one visit for medical attention (OR, 10.15; 95% CI, 3.64-28.32) and pre-existing cardiac/respiratory disease (OR, 3.98; 95% CI, 1.19-13.28). An association of diagnostic delay with inpatient mortality was ascertained specifically in subjects with classic GBS (OR, 5.33; 95% CI, 1.1-25.87). DISCUSSION: Diagnostic delay in GBS results from patient-specific factors and patient pathways. A high index of suspicion is appropriate for certain patient groups. Prospective studies are needed to further investigate this topic.

Acute-onset polyradiculoneuropathy after SARS-CoV2 vaccine in the West and North Midlands, United Kingdom.

INTRODUCTION/AIMS: We aimed to determine whether specific severe acute respiratory syndrome coronavirus 2 (SARS-CoV2) vaccines may be associated with acute-onset polyradiculoneuropathy and if they may result in particular clinical presentations. METHODS: We retrospectively reviewed records of all persons presenting with acute-onset polyradiculoneuropathy from January 1, 2021, to June 30, 2021, admitted to two Neuroscience centers, of the West and North Midlands, United Kingdom. We compared subjects with previous SARS-CoV2 vaccine exposure with a local cohort of persons with acute-onset polyradiculoneuropathy admitted between 2005 and 2019 and compared admission numbers for the studied time frame with that of the previous 3 years. RESULTS: Of 24 persons with acute-onset polyradiculoneuropathy, 16 (66.7%) presented within 4 weeks after first SARS-CoV2 vaccine. Fourteen had received the AstraZeneca vaccine and one each, the Pfizer and Moderna vaccines. The final diagnosis was Guillain-Barré syndrome (GBS) in 12 and acute-onset chronic inflammatory demyelinating polyneuropathy in 4. Among AstraZeneca vaccine recipients, facial weakness in nine persons (64.3%), bulbar weakness in seven (50%), and the bifacial weakness and distal paresthesias GBS variant in three (21.4%), were more common than in historical controls (P = .01; P = .004, and P = .002, respectively). A 2.6-fold (95% confidence interval: 1.98-3.51) increase in admissions for acute-onset polyradiculoneuropathy was noted during the studied time frame, compared to the same period in the previous 3 years. DISCUSSION: Despite a low risk, smaller than that of SARS-CoV2 infection and its complications, exposure to the first dose of AstraZeneca SARS-CoV2 vaccine may be a risk factor for acute-onset polyradiculoneuropathy, characterized by more common cranial nerve involvement.

Retrospective analysis of response to rituximab in chronic inflammatory demyelinating polyneuropathy refractory to first-line therapy.

Few case reports/series describe the efficacy of rituximab in refractory chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), which is preferred in the presence of anti-nodal/paranodal antibodies. We aimed at evaluating the clinical response to rituximab in a subset of patients with refractory CIDP for whom the anti-nodal/paranodal antibodies status was unknown, as not available in Iran. We retrospectively analyzed the response to rituximab in 14 Iranian patients with refractory CIDP (3 children, 11 adults), in whom the anti-nodal/paranodal antibodies status was unknown. The subjects were evaluated with the Medical Research Council (MRC) sum score (MRCSS), Inflammatory Neuropathy Cause and Treatment (INCAT) disability scores, and electrophysiology, before and after treatment. Mean age was 34.4 ± 20.7 years, disease duration pre-rituximab treatment was 27.8 ± 18.8 (range: 6-60) months, and mean follow-up duration was 18.5 ± 11.0 (range: 4-36) months. Considering the INCAT sum score, one worsened during post-rituximab treatment, and three patients did not change. Considering MRCSS, notably, four patients achieved normalization of their MRCSS. Regarding the corticosteroid dose, two patients could discontinue prednisolone. As rated by a pre-defined scoring system, nerve conduction parameters improved significantly post-rituximab in the treated cohort (P = .006). All patients tolerated rituximab infusions without adverse effects. Rituximab may be effective in refractory CIDP, even though worsening may occur in some patients. Anti-nodal/paranodal antibodies assay, when available, and other criteria may help drive therapeutic decision-making on rituximab as second-line treatment.